Study aims to tailor PKU drug dosing for patients with organ impairment
NCT ID NCT07713758
First seen Jul 20, 2026 · Last updated Aug 13, 2026 · Updated 3 times
Summary
This study looks at how the body processes repinatrabit (JNT-517), an oral tablet for phenylketonuria, in people with moderate liver impairment or severe kidney impairment. It compares these results to those from healthy volunteers of similar age, weight, and sex. The goal is to understand if organ impairment changes drug levels in the blood, which could guide future dosing recommendations.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- repinatrabit (JNT-517) oral tablet
- What this could lead to
- If successful, this study could help determine the right dose of repinatrabit for people with phenylketonuria who also have liver or kidney problems.
- What could go wrong
- This is a small, early-phase study focused on drug processing, not on treating the disease. Results may not predict how well the drug works or its safety in larger groups.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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About 32 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jul 2026
- Expected to finish
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Feb 2027
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 65 years
- Sex
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Anyone
- Healthy volunteers
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Accepted
You do not need to have the condition being studied to take part.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion criteria: 1. Body mass index (BMI) from 18.0 to 38.0 kilograms per square meter (kg/m\^2) (inclusive). 2. Able to provide written, informed consent. 3. Male participants and female participants of childbearing potential (POCBP) must agree to remain fully abstinent from intercourse where pregnancy can occur OR practice 2 different contraceptive methods, where at least one must be highly effective. 4. Otherwise be in generally good health. 5. Additional criteria for Group 1: i. Have moderate hepatic impairment determined using Child-Pugh classification (Class B). ii. Have relatively stable hepatic function for the duration of the study as determined by the investigator and liver function test at the time of screening or stable hepatic function 1 year prior to screening. iii. Have normal renal function. iv. Have a clinical diagnosis of hepatic cirrhosis based on biopsy and/or clinical criteria. 6. Additional criteria for Group 2: i. Have estimated glomerular filtration rate based on the Chronic Kidney Disease-Epidemiology Collaboration (eGFRCKD-EPI) lesser than (\<) 30 milliliters per minute per 1.73 square meters (mL/min/1.73 m\^2) indicating severe renal impairment. ii. Not currently be on dialysis. iii. Have relatively stable renal function as determined by eGFRCKD-EPI. 7) Additional criteria for Group 3: i. eGFRCKD-EPI greater than or equal to (≥) 90 mL/min/1.73 m\^2 indicating normal renal function. ii. Have liver enzymes within normal limits. Exclusion criteria: 1. Participants who are breastfeeding. 2. Participants who have a positive pregnancy test result prior to receiving the investigational medical product (IMP). 3. Participants with international normalized ratio (INR) greater than (\>) 2.3. Participants with hepatic impairment with elevated INR will be excluded based on the investigator's discretion. 4. Participants with hepatic carcinoma or porto-hepatic shunts that have been surgically created or planted. 5. A history of difficulty in obtaining intravenous access. 6. The donation of blood or plasma \> 250 milliliters (mL) within 30 days prior to screening or received \> 50 mL of blood or plasma within 30 days prior to screening. 7. Participants who have taken an investigational drug within 30 days prior to screening. 8. Exposure to any substances known to stimulate hepatic microsomal enzymes within 30 days prior to screening (eg, occupational exposure to pesticides, organic solvents, etc). 9. Any participant who, in the opinion of the investigator, should not participate in the trial. 10. Previous exposure to repinatrabit. 11. Additional criteria for Group 1: History of serious mental disorder including participants who have greater than Grade 1 encephalopathy as assessed by the Child-Pugh score and/or the investigator's judgment. 12. Additional criteria for Group 2: i) Participants that have undergone a renal transplant. ii) Participants expected to require dialysis within 45 days. iii) Clinically significant abnormal findings in the serum chemistry, hematology, or urinalysis results obtained at screening or on Day -1, other than those associated with underlying renal conditions and other stable medical conditions consistent with the disease processes. 13. Additional criteria for Group 3: History of or current hepatitis, or carriers of hepatitis B surface antigen (HBsAg) and/or hepatitis C antibodies (anti-HC). Note: Other protocol-specified inclusion/exclusion criteria may apply.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Panax Clinical Research
RECRUITINGMiami, Florida, 33014, United States
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- New pill could tame phenylketonuria by blocking amino acid absorption
- PKU diagnosis: a study on breaking the news to families
- New pill for PKU shows promise in early human testing
- Camp curriculum aims to boost pregnancy outcomes in rare metabolic disorders